PHASE II EFFICACY STUDY OF ROFERON A (R022-8181/002) IN HAIRY CELL LEUKEMIA

ROFERON A (R022-8181/002) 治疗毛细胞白血病的 II 期疗效研究

• 批准号: 3752481
• 负责人: J E JANIK
• 金额: --
• 依托单位: DIVISION OF CANCER TREATMENT
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/3752481
• 关键词: antibody formation; blood disorder chemotherapy; clinical trials; drug administration rate /duration; drug resistance; hairy cell leukemia; human subject; human therapy evaluation; human tissue; immunopathology chemotherapy; interferon alpha; microorganism disease chemotherapy; neoplasm /cancer chemotherapy; neoplasm /cancer remission /regression; neutralizing antibody

This study began as an efficacy study of interferon alpha-2a in patients with hairy cell leukemia. It was observed that most patients responded to interferon, but that very few complete responses were being obtained. Studies being done elsewhere confirmed the low complete remission rate. Once interferon was stopped, nearly uniformly disease progression requiring reinstitution of therapy was observed. There appear to be very few if any patients who will not require further therapy after receiving 12 or 18 months of continuous interferon treatment. Because of these findings, we opted to administer interferon continuously to patients who were initially responsive to this drug. Of the 53 evaluable patients (of the 56 entered on this study), there was one complete remission, 41 partial remissions, 1 minor response, 9 patients with stable disease and only 1 patient with disease progression. Fourteen patients continue to receive interferon without interruption with a median duration of continuous interferon treatment of 9.2 years. Thirty-four patients discontinue interferon for a variety of reasons, the most common being the development of acquired interferon resistance in association with interferon antibodies. The resistance to interferon was manifest early, in the first 18 months of treatment, except in two cases. An important finding in this study is the continued slow, but significant, hematologic improvement in absolute granulocyte and platelet counts beyond 18 months of therapy, thereby indicating that prolonged treatment results in continued benefit rather than the production of antibodies with subsequent development of interferon resistance. Although it is clear from this study that hairy cell leukemia can be controlled in the long-term with interferon, longer follow-up will be necessary to determine if continuous therapy with interferon is better than intermittent therapy.

这项研究最初是一项干扰素 α-2a 对患者的疗效研究 患有毛细胞白血病。据观察，大多数患者对此有反应 干扰素，但很少获得完整的反应。 其他地方进行的研究证实了完全缓解率较低。 一旦停止干扰素，疾病几乎一致进展 观察到需要重新开始治疗。 看来有很 接受治疗后不需要进一步治疗的患者很少（如果有的话） 连续干扰素治疗12或18个月。 因为这些 根据研究结果，我们选择对以下患者持续施用干扰素： 最初对这种药物有反应。在 53 名可评估患者中（ 56 人参加了这项研究），有 1 人完全缓解，41 人 部分缓解，1 例轻微缓解，9 例疾病稳定， 只有 1 名患者出现疾病进展。 十四名患者继续接受治疗 不间断地接受干扰素，中位持续时间为 连续干扰素治疗9.2年。 三十四名患者 由于多种原因停用干扰素，最常见的是 获得性干扰素抵抗的发展与 干扰素抗体。 对干扰素的抵抗很早就表现出来了， 在治疗的前 18 个月内，除两例外。 一个重要的 这项研究的发现是持续缓慢但显着的血液学 18 个月后粒细胞和血小板绝对计数有所改善 治疗，从而表明长期治疗会导致 持续获益而不是随后产生抗体 干扰素耐药性的发展。尽管从这项研究中可以清楚地看出 毛细胞白血病可以长期得到控制 干扰素，需要更长时间的随访以确定是否连续 干扰素治疗优于间歇治疗。